Daniele Canarutto is a Pediatrician at San Raffaele Hospital Pediatric Immunohematology Unit directed by Prof. Alessandro Aiuti and Research Fellow at San Raffaele Telethon Institute for Gene Therapy in Prof. Luigi Naldini’s lab. 

Following a pediatric residency program focused on clinical gene therapy for inborn errors of immunity and metabolism, he received a PhD in Molecular Medicine in 2023, focused on genotoxicity assessment and drug product manufacturing of HDR gene edited CD4 T-lymphocytes for the treatment of Hyper IgM 1. In his work he showed that on-target undesired outcomes are purged out during DP manufacturing and selection of correctly gene edited cells, and developed a large scale IDLV-based manufacturing process to correct CD4 T-cells from Hyper IgM1 patients. He is now coordinating clinical translation for the first in human clinical trial. 

Inspired by this unique setting, whereby correctly edited CD4 T-cells are selected during manufacturing, he designed novel, hematopoietic-stem cell based HDR selection strategies for the treatment of RAG1 deficiency and inborn errors of metabolism, which aim to increase editing safety and efficiency, while also containing costs. These projects are now in advanced stages of preclinical development and we look forward to hear more from them soon.